Guide-specified RNA transcript (N/A)

A guide-specified RNA transcript refers to any RNA molecule, such as messenger RNA (mRNA) or non-coding RNA, that is specifically targeted by a programmable therapeutic system via a complementary guide sequence. This concept is fundamental to RNA-targeting technologies like CRISPR-Cas13, antisense oligonucleotides (ASOs), and small interfering RNA (siRNA), where the drug's specificity is determined by Watson-Crick base-pairing with the target transcript (Abudayyeh et al., 2016, Science). In CRISPR-Cas13 systems, the effector protein is directed to the transcript by a guide RNA (gRNA) to induce site-specific cleavage, effectively silencing gene expression at the post-transcriptional level (Konermann et al., 2018, Cell). This approach is highly versatile, allowing for the treatment of diverse conditions including genetic disorders, viral infections, and cancers by simply redesigning the guide sequence to match the desired transcript (Gootenberg et al., 2017, Science). However, therapeutic development must address significant challenges, including off-target binding to similar RNA sequences and the potential for "collateral" cleavage of non-target bystander RNAs by certain Cas13 orthologs (Abudayyeh et al., 2016, Science). Monitoring the efficacy of such interventions typically involves measuring the reduction in target RNA levels or the resulting protein product (Crooke et al., 2018, Cell Metabolism).

Other names

Target RNARNA targetgRNA-targeted transcriptComplementary RNA sequenceCRISPR-targeted RNA

Programmable RNA-guided binding followed by site-specific cleavage, degradation, or chemical modification of the target transcript to modulate gene expression.